Novartis’ one-time gene therapy for spinal muscular atrophy gets FDA nod

Zolgensma, a gene-based treatment from Novartis (NYSE: NVS) for the treatment of spinal muscular atrophy that affects newborns, received approval from the Food and Drug Administration (FDA). The one-time treatment, which would cost about $2 million as per the current estimates, is touted as a major milestone in the treatment of the often-fatal neuromuscular disorder.

Shares of the Swiss drugmaker gained about 4% on the New York Stock Exchange Friday afternoon, following the news. Novartis CEO Vas Narasimhan in a statement termed the FDA approval a breakthrough in the fight against spinal muscular atrophy. Zolgensma will be offered on the pay-over-time basis, for periods of up to five years.

First Blockbuster Gene Therapy

Considering the hefty price tag, sales might cross $1 billion per year once Zolgansma is launched commercially, which will make it the first blockbuster gene therapy. It will also be the costliest medicine to be ever sold in the US market.

Considering the hefty price tag, sales might cross $1 billion per year once Zolgansma is launched commercially

Sensing the good prospects of new drug development in the fast-growing field of gene therapy, Novartis last year acquired AveXis, the developer of Zolgensma, for $8.7 billion. In the past, the FDA has approved only one gene-therapy for the treatment of inherited health conditions.

Earlier, the company had informed that the cost of Zolgensma would range between a whopping $1.5 million to $5 million. Spinal muscular atrophy, a severe muscle-wasting ailment, is one of the main causes of infant mortality across the world. Due to lack of effective treatment, most of the affected children die before the age of two.

A New Dawn

Hitherto, the only treatment available for spinal muscular atrophy has been Biogen’s (BIIB) Spinraza, which was approved by the FDA a few years ago. Though Spinraza works as a catalyst in the generation of protein in patients, by altering gene expression, it is not considered as a cure.

Also read: Novartis stock falls after Alcon spin-off

Novartis is of the view that the preferred treatment for patients currently under review should be Zolgensma, despite the unprecedented cost. Though the Institute for Clinical and Economic Review (ICER) has approved Zolgensma’s price, in terms of affordability, it recommends a more modest price tag ranging from $1.2 million to $2.1 million.

Novartis shares are currently trading close to $98, the highest value in more than three-and-half years. The stock advanced 17% so far this year and 10% in the past six months.

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